CA3172161A1 - Meganucleases modifiees ayant une specificite pour une sequence de reconnaissance dans le gene d'hydroxyacide oxydase 1 - Google Patents
Meganucleases modifiees ayant une specificite pour une sequence de reconnaissance dans le gene d'hydroxyacide oxydase 1 Download PDFInfo
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- CA3172161A1 CA3172161A1 CA3172161A CA3172161A CA3172161A1 CA 3172161 A1 CA3172161 A1 CA 3172161A1 CA 3172161 A CA3172161 A CA 3172161A CA 3172161 A CA3172161 A CA 3172161A CA 3172161 A1 CA3172161 A1 CA 3172161A1
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- engineered meganuclease
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- C07K2319/09—Fusion polypeptide containing a localisation/targetting motif containing a nuclear localisation signal
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- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Biochemistry (AREA)
- General Health & Medical Sciences (AREA)
- General Engineering & Computer Science (AREA)
- Molecular Biology (AREA)
- Medicinal Chemistry (AREA)
- Biomedical Technology (AREA)
- Biotechnology (AREA)
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- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Enzymes And Modification Thereof (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Abstract
L'invention concerne des méganucléases modifiées qui se lient à une séquence de reconnaissance et clivent une séquence de reconnaissance au sein d'un gène d'hydroxyacide oxydase 1 (HAO1). La présente invention concerne également des méthodes d'utilisation de ces méganucléases modifiées pour fabriquer des cellules génétiquement modifiées. En outre, l'invention concerne des compositions pharmaceutiques comprenant des protéines méganucléases modifiées ou des acides nucléiques codant des méganucléases modifiées selon l'invention et l'utilisation de telles compositions pour le traitement de l'hyperoxalurie primaire de type I (PH1).
Applications Claiming Priority (7)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202163135515P | 2021-01-08 | 2021-01-08 | |
| US63/135,515 | 2021-01-08 | ||
| US202163136097P | 2021-01-11 | 2021-01-11 | |
| US63/136,097 | 2021-01-11 | ||
| US202163241426P | 2021-09-07 | 2021-09-07 | |
| US63/241,426 | 2021-09-07 | ||
| PCT/US2022/011659 WO2022150616A1 (fr) | 2021-01-08 | 2022-01-07 | Méganucléases modifiées ayant une spécificité pour une séquence de reconnaissance dans le gène d'hydroxyacide oxydase 1 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CA3172161A1 true CA3172161A1 (fr) | 2022-07-14 |
Family
ID=80123481
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CA3172161A Pending CA3172161A1 (fr) | 2021-01-08 | 2022-01-07 | Meganucleases modifiees ayant une specificite pour une sequence de reconnaissance dans le gene d'hydroxyacide oxydase 1 |
Country Status (3)
| Country | Link |
|---|---|
| EP (1) | EP4274889A1 (fr) |
| CA (1) | CA3172161A1 (fr) |
| WO (1) | WO2022150616A1 (fr) |
Families Citing this family (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2023133525A1 (fr) | 2022-01-07 | 2023-07-13 | Precision Biosciences, Inc. | Polynucléotides optimisés pour l'expression de protéines |
Family Cites Families (15)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US4873192A (en) | 1987-02-17 | 1989-10-10 | The United States Of America As Represented By The Department Of Health And Human Services | Process for site specific mutagenesis without phenotypic selection |
| US6015832A (en) | 1997-12-31 | 2000-01-18 | The Regents Of The University Of Michigan | Methods of inactivating bacteria including bacterial spores |
| US7767216B2 (en) | 1999-04-28 | 2010-08-03 | The Regents Of The University Of Michigan | Antimicrobial compositions and methods of use |
| US6635676B2 (en) | 1999-04-28 | 2003-10-21 | Regents Of The University Of Michigan | Non-toxic antimicrobial compositions and methods of use |
| US6559189B2 (en) | 1999-04-28 | 2003-05-06 | Regents Of The University Of Michigan | Non-toxic antimicrobial compositions and methods of use |
| US6506803B1 (en) | 1999-04-28 | 2003-01-14 | Regents Of The University Of Michigan | Methods of preventing and treating microbial infections |
| US6555674B2 (en) | 2000-08-09 | 2003-04-29 | Nsgene A/S | JeT promoter |
| US7074596B2 (en) | 2002-03-25 | 2006-07-11 | Board Of Supervisors Of Louisiana State University And Agricultural And Mechanical College | Synthesis and use of anti-reverse mRNA cap analogues |
| US7404969B2 (en) | 2005-02-14 | 2008-07-29 | Sirna Therapeutics, Inc. | Lipid nanoparticle based compositions and methods for the delivery of biologically active molecules |
| ES2735531T3 (es) | 2005-08-23 | 2019-12-19 | Univ Pennsylvania | ARN que contiene nucleósidos modificados y métodos de uso del mismo |
| JP5937292B2 (ja) | 2005-10-18 | 2016-06-22 | デューク大学 | 配列特異性およびｄｎａ−結合親和度が変更された、合理設計メガヌクレアーゼ |
| WO2009001159A1 (fr) | 2007-06-25 | 2008-12-31 | Cellectis | Procédé permettant d'améliorer l'activité de clivage de méganucléases dérivées de i-crei |
| DK2215223T3 (da) | 2007-10-31 | 2013-07-22 | Prec Biosciences Inc | Rationelt konstruerede enkeltkæde-meganucleaser med ikke-palindrome genkendelsessekvenser |
| DK3489366T3 (da) | 2011-06-01 | 2020-02-24 | Prec Biosciences Inc | Fremgangsmåder og produkter til produktion af manipulerede mammale cellelinier med forstærkede transgener |
| EP3898661A1 (fr) * | 2018-12-21 | 2021-10-27 | Precision BioSciences, Inc. | Modification génétique du gène de l'hydroxyacide oxydase 1 pour le traitement de l'hyperoxalurie primaire |
-
2022
- 2022-01-07 WO PCT/US2022/011659 patent/WO2022150616A1/fr unknown
- 2022-01-07 EP EP22701795.1A patent/EP4274889A1/fr active Pending
- 2022-01-07 CA CA3172161A patent/CA3172161A1/fr active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| WO2022150616A1 (fr) | 2022-07-14 |
| EP4274889A1 (fr) | 2023-11-15 |
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